Research for Rare - Research for rare diseases

German network for the research and treatment-optimization of patients with multi-organ autoimmunity (GAIN)

Multi-organ autoimmune diseases belong to the “ultra-rare” disorders and have only recently been recognized being caused by monogenetic mutations in immune-regulatory genes. Patients are typically characterized by multi-organ inflammation including, but not limited to the bone marrow, gut, lungs, kidneys, skin, and the central nervous system (CNS). Genes recently added to the list include CTLA4, LRBA, NFκB1, NFκB2, STAT3, and LAT. This cohort of patients also provides a frontier in medicine, as these rare monogenetic disorders are highly instructive for understanding and treating also more prevalent polygenic autoimmune disorders.

To support those patients, GAIN investigates the underlying molecular and cellular pathomechanisms in multi-organ autoimmune diseases and the molecular interventions that can influence or correct cellular pathology. The aim is to gain a better understanding of pathophysiology in patients with congenital defects of the immune system and to improve treatment.

Projects
Contact

Prof. Dr. med Bodo Grimbacher
Medical Center – University of Freiburg
Institute for Immunodeficiency,
Breisacher Str. 115
79106 Freiburg i. Br.
Phone +49 761 27077-732
Email: bodo.grimbacher@uniklinik.freiburg.de